Local mother-daughter duo lobby government for rare disease strategy

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The rare disease community has launched a petition urging the government to come up with a rare disease strategy that will give patients easier access to lifesaving treatments and medicines.

The Canadian Organization for Rare Disorders (CORD) is calling on all Canadians to come together and call on the government to help save lives by implementing its own rare disease strategy.

CORD had a rare disease strategy already prepared in 2015 but was never adopted. CORD has launched a letter-writing campaign on the Canada4Rare website, encouraging Canadians to write to their local MPs to encourage collaboration between provincial and federal governments in implementing the Canadian Rare Disease Strategy.

The federal government has committed $1 billion to put in place Canada’s first rare disease drug strategy, which was promised to be rolled out this year. But even after two years of planning, the details are not yet finalized.

One of nearly three million Canadians suffers from a rare disease, two-thirds of whom are children. According to CORD, people with a rare disease have more limited access to effective medicines than Canadians with more common conditions and are much less likely to obtain an essential medicine than patients with a rare disease in many other countries.

“We’re trying to build support and get people to understand what’s happening in Canada with rare diseases,” says Beth Vanstone, local rare disease patient advocate.

Beth and her daughter, cystic fibrosis (CF) warrior Madi Vanstone, know this to be true because they have been fighting for the CF community and rare disease patients for a decade to get them the lifesaving medicines they need. they need.

The couple first met then-provincial health critic Christine Elliott nine years ago when they convinced the Ontario government to approve drug coverage for FK Kalyedco, after a long public battle. If Kalydeco had not been funded by OHIP, the Vanstones would have had to pay $350,000 a year for the drug. Even with private insurance and participation in a drug study, they were looking at a cost of about $60,000 per year.

But that was just the beginning of their legacy, as they continue to advocate for others in the rare disease community, who still don’t have access to the medicines they need. They want to know why, in one of the richest countries in the world with a highly developed healthcare system, there are so many red tape and restrictions preventing patients from accessing life-saving drugs. They believe Canada has ignored the rare disease community for too long and now is the time to implement a rare disease strategy similar to countries like the US and UK.

Beth says the rare disease patient plan must be a sustainable method that includes patient and physician engagement to develop workable policies and strategies.

“They (the doctors) are the ones who know the patients and understand the disease, the bureaucrats…don’t understand these rare diseases and how they affect the families of the patients,” Beth said.

According to CORD, Canada remains the only developed country without a national rare disease plan and orphan drug policy. It can take more than seven years for Canadians with a rare disease to be diagnosed, and Canada’s public drug plans provide only 30-40% of rare disease treatments approved by Health Canada.

“We believe there is real commitment and even consensus at the federal and national levels, but there has been relatively little visible dialogue at the provincial government and health system levels,” said Durhane Wong-Rieger, President and CEO of CORD. “We suspect that there are ‘behind the scenes’ conversations between the federal and provincial governments, and these need to be incorporated into the public discourse so that all parities are aligned when this national rare disease drug strategy is Implementation.”

While the Vanstones are aware that rare disease drugs come at a high cost, they believe there are ways to get them into the hands of patients at a fair price.

“However, I think collaboration is the answer to getting these drugs to patients in Canada and I think if Health Canada partners with manufacturers and alongside patients, there can be high cost solutions,” Beth said. .

She also suggested that the cost of treating patients with orphan drugs would economically benefit society as a whole, allowing them to lead healthy lives and contribute to society, instead of filling hospital beds.

She notes that the pandemic has exposed the sour relationship between Health Canada and Big Pharma.

“I hope this opened a lot of people’s eyes to how bad our (healthcare) system is,” Beth said. “Hopefully we can use what happened to highlight all the holes in the system that Canada currently has.”

“I think if Health Canada really wants better for its rare (sick) patients, it needs to change its attitude and start looking for solutions with Pharma,” she said. “And look for different ways to get drugs to patients faster, get them affordable.”

The mother-daughter duo have participated in a number of seminars and webinars in the past, advocating for the rare disease community. The Vanstones will travel to Ottawa in June for a rare disease conference and work on developing the rare disease strategy beginning in 2015.

“We need to do the best we can and hopefully have flexibility for change as we go, because science is changing all the time,” Beth said.

Last summer, the CF drug Trikafta was approved by Health Canada, helping about 1,100 eligible CF patients over the age of 12.

Beth praised Health Minister Christine Elliott for her efforts to get the drug approved for patients in Ontario.

“Trikafta’s struggle really shows how things can happen,” Beth said, noting the long process she had to go through before patients could access it. Some patients may have had faster access due to different coverage approvals in each province. Beth and the rare disease community call it the “postcode lottery,” when only certain patients have access to drugs based on where they live, which they hope a national rare disease strategy will eliminate. She says parts of the lottery problem will be addressed in the country’s Pharamcare plan.

“He shouldn’t spend on where you live whether you have access or not,” Beth said.

It can sometimes take years for drugs to be covered by the provinces after being approved by Health Canada.

“We need something that works for all of Canada,” she said.

On the Canada4Rare website, Canadians will find a petition they can sign on change.org, along with simple steps they can quickly take to let their elected officials know of their support for “Save Rare Lives” and to share the initiative via their social media. platforms.

“I really think it’s important for Canadians to be aware and expect more from our government, so when something happens they’re taken care of too,” she said. “It’s just a matter of putting patients first.”

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